Cited by 130RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryInhalation and Respiratory Drug Delivery

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Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

Guan, Shan, Munder, Antje, Hedtfeld, Silke, Braubach, Peter, Glage, Silke, Zhang, Longgui, Lienenklaus, Stefan, Schultze, Anja, Hasenpusch, Günther, Garrels, Wiebke, Stanke, Frauke, Miskey, Csaba, Johler, Sarah M., Kumar, Yadhu, Tümmler, Burkhard, Rudolph, Carsten, Ivics, Zoltan, Rosenecker, Joseph·2019·Nature Nanotechnology

DOI 10.1038/s41565-018-0358-x

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Abstract

Developing safe and efficient non-viral delivery systems remains a major challenge for in vivo applications of gene therapy, especially in cystic fibrosis. Unlike conventional cationic polymers or lipids, the emerging poloxamine-based copolymers display promising in vivo gene delivery capabilities. However, poloxamines are invalid for in vitro applications and their in vivo transfection efficiency is still low compared with viral vectors. Here, we show that peptides developed by modular design approaches can spontaneously form compact and monodisperse nanoparticles with poloxamines and nucleic acids via self-assembly. Both messenger RNA and plasmid DNA expression mediated by peptide-poloxamine nanoparticles are greatly boosted in vitro and in the lungs of cystic fibrosis mice with negligible toxicity. Peptide-poloxamine nanoparticles containing integrating vectors enable successful in vitro and in vivo long-term restoration of cystic fibrosis transmembrane conductance regulator deficiency with a safe integration profile. Our dataset provides a new framework for designing non-viral gene delivery systems qualified for in vivo genetic modifications.

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Authors: Guan, S., Munder, A., Hedtfeld, S., Braubach, P., Glage, S., Zhang, L., Lienenklaus, S., Schultze, A., Hasenpusch, G., Garrels, W., Stanke, F., Miskey, C., Johler, S. M., Kumar, Y., Tümmler, B., Rudolph, C., Ivics, Z., & Rosenecker, J.
Affiliations: Ludwig-Maximilians-Universität München (DE)
Medizinische Hochschule Hannover (DE)
Ethris (Germany) (DE)
Paul Ehrlich Institut (DE)
GATC Biotech (Germany) (DE)
Corresponding author: Rosenecker, Joseph
ORCID: Guan, Shan
Munder, Antje
Braubach, Peter
Zhang, Longgui
Lienenklaus, Stefan
Garrels, Wiebke
Stanke, Frauke
Miskey, Csaba
Kumar, Yadhu
Tümmler, Burkhard
Rudolph, Carsten
Rosenecker, Joseph
Venue: Nature Nanotechnology
Publisher: Springer Science and Business Media LLC
Published: 2019-01-28
Volume: 14
Issue: 3
Pages: 287-297
ISSN: 1748-3387 (ISSN-L)
1748-3395
DOI: 10.1038/s41565-018-0358-x
OpenAlex: W2914518223
Cited by: 130
References: 51
Citations by year: 2026: 11 · 2025: 21 · 2024: 19 · 2023: 19 · 2022: 21 · 2021: 13 · 2020: 16 · 2019: 10
FWCI: 4.96
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
Locations: Nature Nanotechnology (publishedVersion)
PubMed (publishedVersion)
Funders: Bundesministerium für Bildung und Forschung
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
Keywords: cystic fibrosis, poloxamine, self-assembly, non-viral gene delivery, gene therapy, CFTR, plasmid transfection, mRNA delivery, integrating vectors, in vivo transfection, lung gene delivery
MeSH terms: Amino Acid Sequence, Animals, Cell Line, Cystic Fibrosis, Ethylenediamines, Gene Expression Regulation, Humans, Luciferases, Peptides, RNA, Messenger, Genome, Genes, Reporter, Cystic Fibrosis Transmembrane Conductance Regulator, Transgenes, Mice, Nanoparticles

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Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

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